ArchAngel MLD Trust is a registered charity dedicated to improving the health outcomes of those affected by the rare genetic illness Metachromatic Leukodystrophy.
Metachromatic Leukodystrophy (MLD) is a life-limiting illness which affects around 1 in every 100,000 people. It is caused by a lack of the ARSA enzyme in the patient’s metabolic system and is classified as a ‘demyelinating illness’ (leukodystrophy), a ‘lysosomal storage disorder’ (LSD) and an ‘Inherited Metabolic Disorder’ (IMD).
Supporting Global Medical Teams
We support medical teams worldwide who are working to help people with MLD, providing essential resources, support to clinicians and scientific researchers.
Advocating for Newborn Screening
We are coordinating the application to have MLD added to the UK newborn bloodspot test, ensuring future MLD babies have access to life-saving treatment.
Awarding Grants for UK Families
We award private grants to UK families with children affected by MLD, including for specialist equipment, physical therapies and essential caregiver support.
Advancing Treatments
We promote and support advances in gene therapy and enzyme replacement technologies, providing hope and potential new treatment options for future MLD patients.
ArchAngel MLD Trust was inspired by a little girl named Ava, who was diagnosed with MLD at six years old in 2013. Faced with this devastating diagnosis and no treatment options in the UK, her family sought help globally and discovered the San Raffaele Telethon Institute in Milan, Italy. Ava became the 12th child in the world to participate in a groundbreaking gene therapy trial, which aimed to correct her faulty DNA using her own stem cells. The therapy was successful, altering the predicted course of her disease. Ava’s journey not only brought new hope to her family but also paved the way for the approval and wider use of this life-saving treatment. In 2021 the treatment, now known as ‘Libmeldy’, was approved for use by NHS England, offering a beacon of hope to other families affected by MLD.
Founding and Inspiration
The ArchAngel MLD Trust was founded in 2014 by Ava’s family and family friends in their desire to help others facing this rare and terminal illness.
Trustees
The Trust is an unincorporated association governed by a constitution, Registered Charity Number 1157825. All Trustees are personally connected to MLD and are unpaid volunteers.
Fundraising Goals
The Trust makes grants to organizations engaged in MLD research; awards private grants to individual families and is leading efforts in newborn screening (NBS). We are actively raising funds for our popular grant programme and vital NBS work.
Grants for Families
ArchAngel awards private grants to individual families. ArchAngel has helped numerous UK families purchase wheelchairs, bicycles, breathing apparatus, sensory toys, physiotherapy sessions, home adaptations, and respite care.
Newborn Screening Work
Early diagnosis is crucial, and detecting MLD at birth could save lives. ArchAngel is coordinating the work required to include MLD in the UK’s Newborn Bloodspot (NBS) Screening Programme.